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Biochemistry Structure and function of protein 94387a5c

The first gene therapy (somatic enzyme) was successfully done in

A
SCID
B
Phenylketonuria
C
Thalassemia
D
Cystic fibrosis
High-Yield Explanation
Deficiency of adenosine deaminase, a condition that affects lymphocytes paicularly, was the first disease to be treated by gene therapy. Deficiency of adenosine deaminase accounts for 15% of all cases of severe combined immunodeficiency (SCID)In 1990, Dr. William French Anderson introduced a new copy the gene, carried on a retroviral vector, into a 4-year-old girl suffering from severe combined immunodeficiency (Bubble boy disease).Ref: Harper 30th edition pg: 699

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